What changed in this filing

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Item 7. Management’s Discussion and Analysis

2025-02-27 compared with 2024-02-28 · 319 added, 320 removed, 225 unchanged (74% of the section changed)

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Words such as “may,” “expect,” “anticipate,” “estimate,” “intend,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements.

We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devasting diseases single treatments with potentially curative results.

We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, ALS, refractory MTLE and Fabry disease.

Our internally developed HEMGENIX®, a gene therapy for the treatment of hemophilia B, was approved for commercialization by the FDA in November 2022 and by the EMA in February 2023.

In June 2020, we entered into the Commercialization and License Agreement to license HEMGENIX® to CSL Behring, which is responsible for its commercialization.

We are manufacturing HEMGENIX® for CSL Behring and are entitled to specific milestone payments and royalties on net sales under the Commercialization and License Agreement.

In May 2023, we entered into the Royalty Purchase Agreement for the sale of a portion of the royalty rights due to us from CSL Behring under the Commercialization and License Agreement.

On October 5, 2023, we announced a Reorganization (see definition below) of our research and technology programs.

Business Developments

Below is a summary of our recent significant business developments:

We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results.

We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1, refractory mesial temporal lobe epilepsy (“mTLE”), and Fabry disease.

Huntington’s disease program (AMT-130)

We are currently conducting a multi-center randomized, controlled, and blinded Phase I/II clinical trial for AMT-130 in the U.S.

in which 26 patients with early-manifest Huntington’s disease have been enrolled.

The low-dose cohort of this trial includes 10 patients, of which six patients received treatment with AMT-130 and four patients received imitation surgery.

The high-dose cohort includes 16 patients, of which 10 patients received treatment with AMT-130 and six patients received imitation surgery.

Patients in the high-dose cohort that received imitation surgery had the option to cross over after 12 months if they met the inclusion criteria for the study.

In July 2022, we began crossing over patients in the high-dose cohort who received the imitation surgical procedure.

Four of the six control patients in the high-dose cohort have been crossed over to treatment (three patients received the high dose and one patient received the low dose).

The remaining two control patients in the high-dose cohort did not meet all the inclusion criteria for the study and were not eligible for crossover.

All four crossover patients received a short course of immunosuppression therapy concurrent with the administration of AMT-130.

We are also conducting an open-label Phase Ib/II study in the EU and the United Kingdom, which has enrolled 13 patients with the same early-manifest criteria for Huntington’s disease as the U.S.

Six patients were treated with AMT-130 in the initial low-dose cohort and seven patients were treated in the subsequent high-dose cohort.

In November 2023, we began enrolling patients in a third cohort to further investigate both doses in combination with perioperative immune suppression with a focus on evaluating near-term safety.

Up to 12 patients will be treated in this cohort, all of whom will receive AMT-130 using the current, established stereotactic neurosurgical delivery procedure.

In June 2023, we announced interim data, including up to 24-month follow-up, from 26 patients enrolled in the ongoing U.S.

Phase I/II clinical trial of AMT-130.

In December 2023 we announced updated interim data, including up to 30-month follow-up from the 26 patients enrolled in the ongoing U.S.

Phase I/II clinical trial of AMT-130 as well as the 13 patients enrolled in the European Phase Ib/II trial.

See “ Business —Our Development of AMT-130 for Huntington’s Disease ” for further information on these interim data and the ongoing clinical trials of AMT-130.

Amyotrophic lateral sclerosis program (AMT-162)

On January 31, 2023, we announced that we had entered into a global licensing agreement with Apic Bio for a one-time, intrathecally administered investigational gene therapy for ALS caused by mutations in SOD1.

We made an initial cash payment in February 2023 of $10.0 million to Apic Bio that was recognized as a research and development expense.

We owe up to $43.0 million in milestone payments to Apic Bio if AMT-162 is approved for commercialization in the U.S.

Temporal lobe epilepsy program (AMT-260)

AMT-260 is our gene therapy candidate for refractory MTLE.

We acquired AMT-260 through our acquisition of uniQure France SAS in July 2021.

In September 2023, following the clearance of an IND for AMT-260 in August 2023, we paid EUR 10.0 million ($10.6 million) to the former shareholders of uniQure France SAS, to settle a milestone payment owed in relation to our 2021 acquisition.

Reorganization

On October 5, 2023, we announced that we are implementing a Reorganization.

As a result of the Reorganization, we discontinued investments in more than half of our research programs, including AMT-210 for the treatment of Parkinson’s disease and certain technology projects.

Following the Reorganization, we are prioritizing advancing our clinical-stage programs to clinical proof of concept.

As a result of the Reorganization, we eliminated approximately 20% of our total workforce and closed our research laboratory in Lexington, Massachusetts.

We completed the Reorganization by the end of fiscal year 2023 and incurred costs of approximately $2.5 million for cash expenditures related to employee severance costs.

We also incurred costs associated with the closure of a research laboratory in Lexington and associated fixed assets of which the carrying values were determined to not be recoverable as of the cease-use date in November 2023.

As part of the Reorganization, we also consolidated all GMP manufacturing into our Lexington manufacturing facility and consolidated process and analytical development into our Amsterdam, Netherlands facility.

As a result of the significant reduction in research activities, Ricardo Dolmetsch, Ph.D., departed as Chief Scientific Officer, effective October 4, 2023.

Dolmetsch served as a scientific consultant through the end of the year.

In connection with Dr.

Dolmetsch’s transition, the Board of Directors of the Company have appointed Richard Porter, Ph.D., our current Chief Business Officer, to serve as the Company’s Chief Business and Scientific Officer, effective as of October 5, 2023.

AMT-130 is our novel gene therapy candidate for the treatment of Huntington’s disease, which utilizes our proprietary, gene-silencing miQURE platform and incorporates an AAV vector carrying a micro ribonucleic acid (“miRNA”) specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.

We are currently conducting the U.S.

and European studies.

We completed the enrollment of all 26 patients in the first two cohorts of our US study in March 2022 and the enrollment of 13 patients in the two cohorts of our European study in June 2023.

As of February 2025, we have enrolled 12 patients into a third cohort to further investigate both doses of AMT-130 together with perioperative immunosuppression using the current, established stereotactic administration procedure.

RMAT Designation

In June 2024, we announced that the FDA granted us RMAT designation for AMT-130.

Updated Interim Data

In July 2024, we announced updated interim clinical data, including up to 24 months of follow-up data from 29 patients enrolled in the U.S.

study and the European study as of a March 31, 2024 cut-off date.

Regulatory Alignment

In December 2024, following our initial Type B meeting, we announced that we had reached agreement with FDA on key elements of an accelerated approval pathway for AMT-130.

Other clinical candidates

In November 2024, we announced that the that the first patient was dosed in the GenTLE Phase I/IIa clinical trial of AMT-260 for the treatment of refractory mTLE.

In addition, the FDA recently approved a protocol amendment expanding the inclusion criteria for certain patients in the first cohort to include patients with non-lesional mesial temporal lobe epilepsy in the non-dominant hemisphere.

In August 2024, we announced that the first patient was dosed in a Phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease.

In February 2025, we announced that we completed the enrollment of the first cohort of the clinical trial and that we would proceed enrolling the second cohort of the clinical trial.

In October 2024, we announced the first patient was dosed in a Phase I/IIa clinical trial of AMT-162 for the treatment of SOD1-ALS.

In January 2025, we announced that we completed the enrollment of the first cohort of the clinical trial and that we would proceed enrolling the second cohort of the clinical trial.

Sale of commercial manufacturing activities

In June 2024, we and Genezen entered into the Lexington Transaction, which closed in July 2024.

Genezen acquired manufacturing facility including equipment and related manufacturing operations with a carrying value of $15.2 million, inventory with a carrying value of $8.8 million and certain other assets (including allocated goodwill) with a carrying value of $2.8 million associated with the Lexington Facility on Closing.

As consideration for the Lexington Transaction, we received from Genezen (i) shares of newly issued Series C preferred stock of Genezen Holdings Inc.

valued at $12.5 million, which are convertible into common stock and will accrue an 8.0% per annum cumulative dividend, (ii) a convertible promissory note with a nominal amount of $12.5 million, bearing interest at 8.0% per annum and maturing 63 months following the date of issuance valued at $13.3 million and (iii) a right to purchase HEMGENIX® at terms considered favorable to market terms (the “Consideration”) valued at $16.7 million.

We also included a firm purchase commitment liability, valued at $8.8 million for our customer contract with CSL Behring inherently related to the right to purchase HEMGENIX® in the consideration received.

We recorded a $1.2 million gain from the divestment.

We paid a total of $8.3 million to Genezen and CSL Behring related to adjustments of working capital and to obtain consent to proceed with the divestiture.

In connection with the Closing, we, Genezen and the landlord of the Lexington Facility entered into an agreement for us to assign and Genezen to assume the existing lease agreement between us and the landlord.

We also amended our original July 2013 guarantee to continue guaranteeing rental payments owed by Genezen until the end of the current term on May 31, 2029.

In the event of Genezen’s default related to rental payments owed to the landlord, we are entitled to terminate the assignment agreement and step into the original lease agreement.

At the Closing, we entered into a commercial supply agreement (“CSA”) with Genezen.

Pursuant to the CSA, Genezen manufactures and supplies CSL Behring’s commercial demand for HEMGENIX® on our behalf.

The CSA includes a minimum term of three years and minimum purchase commitments of HEMGENIX® commercial supplies of $43.3 million over the first three years, unless certain contractual provisions are triggered.

Our obligations with respect to the supply of HEMGENIX® to CSL Behring remain in effect notwithstanding the subcontracting to Genezen.

Organizational restructuring

In August 2024, we announced the Restructuring to conserve capital and streamline the organization.

As a result of the Restructuring and Lexington Transaction, we eliminated approximately 300 positions or 65% of our workforce.

We substantially completed the Restructuring at the end of 2024.

We have incurred $5.2 million of employee severance costs in the year ended December 31, 2024 and estimate that we will incur additional costs in the range of $0.5 million to $1.0 million in 2025.

Royalty financing agreement

On May 12, 2023, we entered into the Royalty Financing Agreement with the Purchaser.

In May 2023, we entered into a royalty purchase agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P.

(the “Purchaser”).

Under the terms of the Royalty Financing Agreement, we received an upfront payment of $375.0 million in exchange for the Purchaser’s rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX® for certain current and future royalties due to us.

We are also eligible to receive an additional $25.0 million milestone payment under the Royalty Financing Agreement if 2024 net sales of HEMGENIX® exceed a pre-specified threshold.

The Purchaser will receive 1.85 times the upfront payment (or $693.8 million) and 1.85 times the $25.0 million milestone payment (if paid) prior to the First Hard Cap Date or, if such cap is not met, up to 2.25 times the upfront and milestone payment (if paid) through December 31, 2038.

If 2024 net sales do not exceed a pre-specified threshold, we will be obligated to pay $25.0 million to the Purchaser but only to the extent that we achieve a future sales milestone under the CSL Behring Agreement.

If such milestone payment is not due from CSL Behring, we are not obligated to pay any amounts to the Purchaser.

We will be obligated to pay $25.0 million of the first worldwide sales milestone payment from CSL Behring, if received, to the Purchaser.

The Purchaser will receive 1.85 times the upfront payment (or $693.8 million) until June 30, 2032 (“First Hard Cap Date”) if such thresholds are met or, if such cap is not met by June 30, 2032, up to 2.25 times of the upfront payment through December 31, 2038 (“Second Hard Cap Date”).

If, on or prior to the defined dates for each cap amount, the total amount of royalty payments received by the Purchaser equals or exceeds the cap amount applicable to such date, the Royalty Financing Agreement will automatically terminate and all rights to the HEMGENIX® royalty payments will revert back to us.

We have no obligation to repay any amounts received from the Purchaser in the event that the applicable cap amount is not reached during the term of the Royalty Financing Agreement.

As of December 31, 2024 we expect to satisfy our commitment to the Purchaser prior to the Second Hard Cap Date.

Until December 2024, we assumed that we would satisfy the commitments to the Purchaser prior to the First Hard Cap Date.

We retained the rights to all other royalties, as well as contractual milestones totaling up to $1.3 billion, under the terms of the CSL Behring Agreement.

Hercules amendment

Upon entering into the Royalty Financing Agreement, we and Hercules amended the 2021 Restated Facility on May 12, 2023.

The 2023 Amended Facility extends the maturity date and interest-only period from December 1, 2025 to January 5, 2027.

Investment in debt securities

In July and September 2023, we invested $272.0 million and EUR 87.0 million (or a total of $366.4 million as of the investment dates) of our cash and cash equivalents into short-term U.S.

and European government bonds that are U.S.

dollar and euro denominated, respectively.

Our investment policy requires us to invest in bonds with the highest investment grade credit rating.

As of December 31, 2023, the bonds have remaining maturities ranging from less than one month to seven months.

We classify these bonds as held-to-maturity.

CSL Behring collaboration

In June 2023, the first sale of HEMGENIX ® in the U.S.

occurred, and in July 2023 we collected the $100.0 million owed to us under the CSL Behring Agreement.

Hercules loan repayment and amendment

Upon entering into the Royalty Financing Agreement in May 2023, we and Hercules amended our existing term loan facility.

Through the May 2023 amendment we extended the maturity date and interest-only period from December 1, 2025 to January 5, 2027 (the “Maturity Date”).

In connection with the Closing we once more amended the facility.

As a condition to Hercules’ consent to the Lexington Transaction we prepaid $50.0 million of the total $100.0 million principal outstanding in July 2024.

The remaining $50.0 million principal outstanding will need to be repaid at the Maturity Date.

In January 2025, we raised $70.1 million of net proceeds, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 4.4 million ordinary shares at a price to the public of $17.00 per ordinary share.

In February 2025, we raised an additional $10.6 million in net proceeds upon the underwriters’ exercise of their option to purchase an additional 0.7 million ordinary shares at the public offering price.

Financial Highlights

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Selling, general and administrative expenses

Net (loss) / income

As of December 31, 2024, we had $367.5 million in cash and cash equivalents and investment securities (December 31, 2023:

$617.9 million cash and cash equivalents and investment securities).

We had a net loss of $308.5 million in 2023, a net loss of $126.8 million in 2022 and net income of $329.6 million in 2021.

We had a net loss of $239.6 million in 2024 and a net loss of $308.5 million and $126.8 million in 2023 and 2022, respectively.

As of December 31, 2024, we had an accumulated deficit of $1,130.0 million (December 31, 2023:

$890.4 million).

Compared to the year ended December 31, 2023, we expect that our research and development expenses will decline following the Reorganization, until such time we decide to advance one of our gene therapy product candidates into late-stage clinical development.

See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.

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We also discuss our critical accounting estimates with the Audit Committee of our Board of Directors.

We consider the following to be our critical accounting estimate:

We consider the following to be our critical accounting estimates in the year ended December 31, 2024:

● Contingent consideration recorded in relation to the uniQure France SAS business combination;

● Valuation of the intangible asset with favorable terms recorded as part of the Lexington Transaction;

● Date at which we expect to satisfy the commitment under the Royalty Financing Agreement.

Contingent consideration

On the Acquisition Date of uniQure France SAS we recorded contingent consideration related to amounts potentially payable to uniQure France SAS’s former shareholders.

The amounts payable in accordance with the SPA are contingent upon realization of certain milestones associated with the TLE research program.

In July 2021 (“Acquisition Date”), we acquired uniQure France SAS (“Acquisition Date”) and we recorded contingent consideration related to amounts potentially payable to uniQure France SAS’s former shareholders.

The amounts payable in accordance with the share and purchase agreement (“SPA”) are contingent upon realization of certain milestones associated with AMT-260.

Contingent consideration was measured at fair value at the Acquisition Date with changes in fair value recognized in the consolidated statements of operations in research and development expenses.

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● We had used discount rates ranging from 15.3% to 15.6% to calculate the contingent consideration as of December 31, 2023.

As of December 31, 2023 we increased the interest rates to a range of 15.3% to 15.6% to reflect increases in market interest rates.

As of December 31, 2024 we used discount rates ranging from 15.3% to 16.2% to reflect increases in market interest rates.

An increase in the discount rate reduces the fair value of the contingent consideration liability whereas a decrease in the discount rate increases the fair market value of the contingent consideration liability.

● We need to develop an estimate of when a milestone is expected to be achieved.

● We need to regularly update our estimate of when a milestone is expected to be achieved.

An achievement of a milestone at a later than currently expected date reduces the fair value of the contingent consideration liability whereas an achievement at an earlier than currently expected date increases the fair value of the contingent consideration liability.

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During the year ended December 31, 2023 we increased the probability to 100.0% following the clearance of the IND application for AMT-260 in August 2023.

We did not change the probabilities of any further milestones following the dosing of the first patient in a Phase I/II clinical trial in November 2024.

The increase in probabilities resulted in a $14.2 million expense in the year ended December 31, 2023 and a $4.4 million expense in the year ended December 31, 2022.

This also resulted in an increase of the probability that AMT-260 may advance to late-stage development and commercialization.

The fair value of the contingent consideration liability as of December 31, 2023 was $43.0 million and as of December 31, 2022 was $35.3 million.

The increase was primarily driven by the increase in the probability of TLE advancing into clinical development to 100% following the clearance of the IND application for AMT-260 in August 2023.

If as of December 31, 2023 we had assumed TLE was certain (i.e., 100% probability) to advance into late-stage development and commercialization, then the fair value of the contingent consideration liability would have increased from $43.0 million to $75.9 million.

If as of December 31, 2023 we had assumed that we would discontinue development of the TLE program, then we could have released the contingent consideration liability to income.

In prior periods, we have considered the following to be critical accounting estimates but have determined that these are no longer considered critical for the current year:

● Valuation allowance related to Dutch deferred tax assets;

● Revenue recognition related to CSL Behring milestones

Valuation allowance related to Dutch deferred tax assets

We are subject to corporate taxes in the Netherlands.

We have been incurring net operating losses in accordance with the corporate tax laws in almost all years since we founded our business.

As of December 31, 2022, the total amount of net operating losses carried forward under the Dutch tax regime was $264.0 million (December 31, 2021:

$228.5 million).

We have historically recorded a full valuation allowance.

We evaluate all positive and negative evidence including future income from the CSL Behring Agreement in assessing the need for such a full valuation allowance.

We concluded that as of December 31, 2022 it is more likely than not that the remaining deferred tax assets will not be realized.

If we would have released the full valuation allowance as of December 31, 2022, then we would have recorded up to $74.5 million of deferred tax income during the year ended December 31, 2022.

This is no longer considered a critical accounting estimate as future income from the CSL Behring Agreement is not expected to result in a taxable profit.

Revenue recognition related to CSL Behring milestones

On June 24, 2020 (“ Signing Date”), we entered into the CSL Behring Agreement.

The transaction became effective on Closing.

As of Closing, we identified two material performance obligations related to the CSL Behring Agreement:

(i) Sale of the exclusive global rights to the Product (“License Sale”);

(ii) Generate information to support the regulatory approval of the current and next generation manufacturing process of Product and to provide any such information generated to CSL Behring (“Manufacturing Development”).

We determined that the fixed upfront payment of $450.0 million and the $12.4 million that we received in relation to the certain reimbursable activities to fulfill the transfer of global rights (“Additional Covenants”) in the year ended December 31, 2021 should be allocated to the License Sale.

In addition, we concluded that $255.0 million of variable milestone payments, sales milestone payments and royalties should be allocated to the License Sale performance obligation as well.

We determined that the License Sale was completed on May 6, 2021, when we transferred the license and CSL Behring assumed full responsibility for the development and commercialization of the Product.

Upon the Closing, we evaluated the amounts of potential payments and the likelihood that the payments will be received.

We utilized the most likely amount method to estimate the variable consideration to be included in the transaction price.

Since we cannot control the achievement of regulatory and first commercial sales milestones, we concluded that all potential payments were constrained as of Closing.

We determined that we would recognize revenue related to these payments, only to the extent that it becomes probable that no significant reversal of recognized cumulative revenue will occur thereafter.

We will include payments related to sales milestones in the transaction price when their achievement becomes probable, and we will include royalties on the sale of Product once these have been earned.

In March and April 2022, we collected the $55.0 million of variable milestone payments related to the submissions of a BLA and MAA which we had recorded as license revenue in the year ended December 31, 2021.

During the year ended December 31, 2022, we recorded $100.0 million of variable milestone revenue related to a first sale of HEMGENIX™ and collected this payment in July 2023.

We no longer consider this a critical accounting estimate because we currently do not expect to recognize any regulatory milestone payments within the next two years.

The recognition of license revenue related to sales milestone payments and royalties does not require significant judgement as we recognize these as sales of HEMGENIX™ occur.

The fair value of the contingent consideration liability as of December 31, 2024 was EUR 10.5 million ($10.9 million) and as of December 31, 2023 was EUR 39.0 million ($43.0 million).

The reduction was primarily driven by a EUR 26.8 million ($28.2 million) milestone payment we made in December 2024 related to the first patient dosed in the AMT-260 clinical trial.

If as of December 31, 2024, we had assumed a 100% likelihood of AMT-260 advancing into a Phase III clinical study, then the fair value of the contingent consideration would have increased to EUR 33.7 million ($35.0 million).

If as of December 31, 2024 the Company had assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would have been released to income.

Intangible asset

Part of the consideration received in the Lexington Transaction included a right, with an estimated fair market value of $16.7 million, to purchase HEMGENIX® from Genezen up to the termination of our supply agreement with CSL Behring at terms considered favorable to market terms.

The determination of the fair value required us to estimate the current and future commercial supply prices of HEMGENIX® to compare these to the contractually agreed terms at Closing.

Prices for the commercial supply of HEMGENIX® are not readily observable in the market.

Therefore, our estimate of the commercial supply price was based on what a market participant would be willing to pay in a comparable transaction, using industry benchmarks, relevant market data, current and historical commercial supply agreements, as well as historical production cost information as reference points.

If the estimated commercial supply price used in the valuation had increased by 5% per drug product batch, then the fair market value of the intangible asset and the consideration received as well as the gain recorded on the divestment would have been increased by $3.0 million.

If the estimated commercial supply price used in the valuation had been decreased by 5% per drug product batch, then the fair market value of the intangible asset and the consideration received on the divestment would have been decreased by $2.9 million.

This would have resulted in a loss on divestment of $1.7 million instead of the $1.2 million gain presented.

Date at which we expect to satisfy the commitment under the Royalty Financing Agreement

On May 12, 2023, we entered into the Royalty Financing Agreement with the Purchaser.

Under the terms of the Royalty Financing Agreement we received an upfront payment of $375.0 million in exchange for its rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX® for certain current and future royalties due to us (refer to Note 13 “Royalty Financing Agreement” for further details).

Pursuant to the agreement, the total amount of royalties to be received by the Purchaser is subject to an increasing cap equal to (i) $693.8 million up to the First Hard Cap Date if such thresholds are met or, if such cap is not met by the First Hard Cap Date (ii) $843.8 million through the Second Hard Cap Date.

We have no obligation to repay any amounts received from the Purchaser in the event that the applicable cap amount is not reached during the term of the Royalty Financing Agreement.

As of December 31, 2024, we expect to satisfy our commitment to the Purchaser prior to the Second Hard Cap Date.

Until December 2024, we assumed that we would satisfy the commitments to the Purchaser prior to the First Hard Cap Date.

As a result of this change in estimate we expect that our total payments of interest expense will increase from $318.8 million by $150.0 million to $468.8 million over a term that was extended from 9 years to 15.5 years.

Revenue recognition related to CSL Behring variable milestone payments

We sold the exclusive global rights HEMGENIX™ to CSL Behring in 2021.

The consideration received included variable milestone payments which we recorded as revenue once probable.

We recorded $100.0 million of variable milestone revenue related to a first sale of HEMGENIX™ as license revenue in the year ended December 31, 2022 following the November 2022 BLA approval.

We collected the payment in July 2023.

We do not consider this to be a critical accounting estimate for the years ended December 31, 2024 and 2023 because we currently do not expect to recognize any regulatory milestone payments within the next 12 months.

Recently Adopted Accounting Pronouncements

In the year ended December 31, 2024, we adopted Accounting Standards Update (ASU) 2023-07, Segment Reporting (Topic 280):

Improvements to Reportable Segment Disclosures (“ASU 2023-07”).

This update requires us to disclose segment expenses that are significant and regularly provided to our chief operating decision maker (“CODM”).

In addition, ASU 2023-07 requires us to disclose the title and position of its CODM and how the CODM uses segment profit or loss information in assessing segment performance and deciding how to allocate resources.

The adoption of this update did not have a material impact on our financial position or results of operations but resulted in expanded disclosures in the segment reporting section of the financial statements.

We adopted ASU 2023-07 using a retrospective transition method.

Results of Operations

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Operating expenses:

Cost of revenues

Cost of license revenues

Cost of contract manufacturing

Research and development expenses

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Other expense

(Loss) / income from operations

Non-operating (expense) / income, net

(Loss) / income before income tax benefit

Loss from operations

Non-operating expense, net

Loss before income tax benefit

Income tax (expense) / benefit

Net (loss) / income

Revenues and cost of revenues

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Cost of contract manufacturing revenues

Effective on Closing of the CSL Behring Agreement we sold the exclusive global rights to the Product (“License Sale”).

We recognize license revenue in relation to the License Sale when it becomes probable that regulatory and sales milestone events will be achieved as well as when royalties on sales of Product have been earned.

We sold the exclusive global rights to HEMGENIX® to CSL Behring in 2021 (“License Sale”).

We recognized $10.1 million, $2.8 million and $100.0 million of license revenue for the years ended December 31, 2024, 2023 and 2022, respectively.

We recognized $2.8 million of license revenue in 2023 related to royalty payments owed on HEMGENIX® sales, when earned.

We recognized $100.0 million of license revenue in 2022 related to a milestone payment following the first sale of HEMGENIX ® in the U.S.

in 2023, which we considered probable as of December 31, 2022.

We recognized $517.4 million license revenue in 2021 related to the fixed upfront payment of $450.0 million and an additional payment of $12.4 million we received after the Closing as well as a total of $55.0 million of payments related to milestone payments owed on submission of the MAA in March 2022 and the BLA in April 2022, which we had considered probable as of December 31, 2021.

We expense contract fulfillment costs associated with license revenue we receive from CSL Behring, which are recognized as costs of license revenues.

These expenses primarily consist of payments we owe to our licensors in relation to license payments we receive from CSL Behring.

We recognized $10.1 million and $2.8 million of license revenue in 2024 and 2023 related to royalty payments owed on HEMGENIX® sales, when earned.

We recognized $100.0 million of license revenue in 2022 related to a milestone payment owed on the first sale of HEMGENIX ® in the U.S.

In 2008, we entered into a license agreement with St.

Jude Children’s Research Hospital (“St.

Jude”), which we amended in 2012.

Under this license agreement, St.

Jude has granted us an exclusive license, with a right to sublicense, to patent rights relating to expression of hFIX in gene therapy vectors, to make, import, distribute, use, and commercialize products containing hFIX covered by a valid patent claim in the field of gene therapy for treatment or prophylaxis of hemophilia B.

The majority of the U.S.

patent rights will expire in 2028 and the European patent rights (along with one U.S.

patent) will expire in 2025.

With respect to our collaboration with CSL Behring, we have agreed with St.

Jude on an apportionment of certain amounts we receive from CSL Behring as sublicensing revenue that is equivalent to a low-single digit percentage of such amounts.

The agreement will remain in effect until no further payment is due relating to any licensed product under this agreement or either we or St.

Jude exercise our rights to terminate it.

Jude may terminate the agreement in specified circumstances relating to our insolvency.

We may terminate the agreement for convenience at any time subject to a specified notice period.

We treat license payments to St.

Jude as contract fulfillment costs associated with license revenue we receive from CSL Behring and recognize these as costs of license revenues.

We incurred $1.3 million, $0.1 million and $1.3 million of such cost in the years ended December 31, 2024, 2023 and 2022, respectively.

We recognize collaboration revenues associated with services to CSL Behring in accordance with the CSL Behring Agreement.

Collaboration revenue related to these contracted services is recognized when the performance obligations are satisfied.

We recognize collaboration revenues associated with services we provide to CSL Behring.

Collaboration revenue is recognized when the performance obligations are satisfied.

We recognized $10.9 million, $2.3 million and $3.0 million of collaboration revenue for the years ended December 31, 2024, 2023 and 2022, respectively.

The decrease in collaboration revenue in 2023 of $0.7 million compared to 2022 was primarily related to a reduction in services requested by CSL Behring following the submissions of the BLA and MAA for HEMGENIX ®.

The increase in collaboration revenue in 2022 of $0.6 million compared to 2021 was primarily related to revenues related to full-time-employee (“FTE”) recharges of $3.0 million recognized from the CSL Behring Agreement as a result of additional development services that CSL Behring requested from us.

We recognize contract manufacturing revenues related to contract manufacturing HEMGENIX ® for CSL Behring.

Contract manufacturing revenues are realized when earned upon sales of HEMGENIX ® to CSL Behring.

We recognized $10.8 million and $1.7 million contract manufacturing revenues in the year ended December 31, 2023 and 2022, respectively.

We did not recognize such revenues in 2021 as we started contract manufacturing activities to supply CSL Behring with HEMGENIX ® following their submission of a BLA and MAA in the spring of 2022.

We incurred $13.6 million and $2.1 million of cost of contract manufacturing revenues related to the manufacture of HEMGENIX® in the years ended December 31, 2023 and 2022 respectively, compared to nil cost of contract manufacturing revenues in the year ended December 31, 2021.

Costs of contract manufacturing revenues has increased in the year ended December 31, 2023 compared to the year ended December 31, 2022 due to the increase in sales of HEMGENIX® and a write-down of inventory that has a cost basis in excess of its expected net realizable value.

We recognized collaboration revenues associated with certain pre-clinical analytical development and process development activities that were reimbursable to us by Bristol-Myers Squibb (“BMS”) under the initial collaboration, research and license agreements (“BMS CLA”) and the amended agreements (“amended BMS CLA”) as well as other related agreements.

Collaboration revenue related to these contracted services were recognized when performance obligations were satisfied.

We recognized nil, $1.8 million and $4.2 million of collaboration revenue for the years ended December 31, 2023, 2022 and 2021, respectively.

Following the termination of the amended BMS CLA on February 22, 2023 we did not recognize any further revenue for services rendered in accordance with the BMS CLA.

The increase in collaboration revenue in 2024 of $8.6 million compared to 2023 was primarily related to additional development and other services provided in relation to the transfer of manufacturing responsibility to CSL Behring.

We provided contract manufacturing service to CSL Behring between April 2022 and July 2024.

We transferred these activities to Genezen as part of the Lexington Transaction.

Following the Closing of the Lexington Transaction, title to HEMGENIX® supply directly passes from the contract manufacturer, Genezen, to CSL Behring.

We do not control HEMGENIX® before it is transferred to CSL Behring.

We arrange for HEMGENIX® to be provided by Genezen to CSL Behring.

We determined that we are an agent in the sale of HEMGENIX® to CSL Behring.

As a result of being an agent, we recognize corresponding costs related to the purchase of HEMGENIX® from Genezen net of income from the sales of HEMGENIX® to CSL Behring and income related to the release of liabilities associated with expected net losses in Other expense within the Company’s Consolidated Statements of Operations and Comprehensive Loss.

We recognized contract manufacturing revenues related to contract manufacturing HEMGENIX ® for CSL Behring when earned upon sales of HEMGENIX ® to CSL Behring.

We recognized $6.1 million, $10.8 million and $1.7 million contract manufacturing revenues in the years ended December 31, 2024, 2023 and 2022, respectively.

We incurred $17.1 million of cost of contract manufacturing revenues related to the manufacture of HEMGENIX® in the year ended December 31, 2024 and $13.6 million and $2.1 million of cost of contract manufacturing revenues, in the years ended December 31, 2023 and 2022 respectively.

Costs of contract manufacturing revenues increased in the year ended December 31, 2024 compared to the year ended December 31, 2023 primarily due to expensing costs that could not be recovered from selling HEMGENIX ® under the terms of our development and commercial supply agreement with CSL Behring.

Costs of contract manufacturing revenues increased in the year ended December 31, 2023 compared to the year ended December 31, 2022 due to the increase in sales of HEMGENIX® and a write-down of inventory that had a cost basis in excess of its expected net realizable value.

Following the termination of the collaboration, research and license agreements and the amended agreements with Bristol-Myers Squibb (“BMS”) in February 2023, we did not recognize any further revenue for services rendered.

We recognized nil collaboration revenue for the years ended December 31, 2024 and 2023 and $1.8 million for the year ended December 31, 2022.

Res e arch and development expenses

6 unchanged sentences

● costs incurred for the development and improvement of our manufacturing processes and methods;

● costs associated with research activities for enabling technology platforms, such as next-generation vectors, promoters and re-administration of gene therapies;

● costs associated with research activities for enabling technology platforms;

● costs associated with the rendering of collaboration services;

● payments related to identifiable intangible assets without an alternative future use;

● payments to our licensors for milestones that have been achieved related to our product candidates, including approval of the MAA and BLA for HEMGENIX ® ;

● payments to our licensors for milestones that have been achieved related to our product candidates;

● facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies;

2 unchanged sentences

● AMT-130 (Huntington’s disease).

We have incurred costs related to preclinical and nonclinical studies of AMT-130 and have been incurring costs related to our U.S.

Phase I/II clinical trial since February 2019.

Since 2021, we have also incurred costs related to our Phase Ib/II clinical trial in Europe;

We have been incurring costs related to our U.S.

study since February 2019.

Since 2021, we have also incurred costs related to our European study;

● AMT-260 (Temporal lobe epilepsy) .

We have incurred costs related to the preclinical development of AMT-260.

We acquired this program on July 30, 2021;

We have incurred costs related to the development of AMT-260.

We acquired this program in July 2021;

● AMT-191 (Fabry disease) .

We have incurred costs related to the preclinical development of AMT-191 for the treatment of Fabry disease;

● AMT-162 ( Amyotrophic Lateral Sclerosis caused by mutations in SOD1 ) We have incurred costs related to the acquisition in addition to costs incurred to initiate a Phase I/II clinical trial;

● Etranacogene dezaparvovec (hemophilia B) .

We have incurred costs related to the research, development, and production of etranacogene dezaparvovec for the treatment of hemophilia B.

Up to the Closing of the CSL Behring Agreement, we incurred costs related to the preparation of a BLA and MAA and for commercialization of the Product.

We also incurred costs for manufacturing development.

After the Closing, CSL Behring is responsible for the clinical and regulatory development and commercialization of the Product;

We have incurred costs related to the development of AMT-191 for the treatment of Fabry disease;

● AMT-162 ( Amyotrophic Lateral Sclerosis caused by mutations in SOD1 ) We have incurred costs related to the acquisition in addition to costs incurred to initiate and conduct a Phase I/II clinical trial;

● AMT-061 (Hemophilia B) .

We transitioned activities related to the clinical trial and long-term follow-up of patients to CSL Behring in December 2022.

Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the periods ended December 31, 2023 and 2024 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition.

● Preclinical research programs .

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● Technology platform development and other related research .

We incurred significant research and development costs related to manufacturing and other enabling technologies that are applicable across all our programs.

Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including manufacturing campaigns, regulatory submissions, and enrollment of patients in clinical trials.

We incurred significant research and development costs related to manufacturing prior to the Closing of the Lexington Transaction and other enabling technologies that are applicable across all our programs.

Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including regulatory submissions, and enrollment of patients in clinical trials.

The successful development of our product candidates is highly uncertain.

1 unchanged sentence

● the scope, rate of progress and expense of our research and development activities;

● our ability to successfully manufacture and scale-up production;

● clinical trial protocols, speed of enrollment and resulting data;

7 unchanged sentences

(in thousands)

Amyotrophic lateral sclerosis (AMT-162)

Temporal lobe epilepsy (AMT-260)

Huntington's disease (AMT-130)

Temporal lobe epilepsy (AMT-260)

Amyotrophic lateral sclerosis (AMT-162)

Fabry disease (AMT-191)

Etranacogene dezaparvovec (AMT-060/061)

Programs in preclinical development and platform related expenses

Hemophilia B (AMT-061)

Total direct research and development expenses

2 unchanged sentences

Share-based compensation expense

Severance costs

Impairment charge

Fair value changes related to contingent consideration

Other expenses

Impairment related to the Reorganization

Total other research and development expenses

1 unchanged sentence

Direct research and development expenses

Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)

On January 31, 2023, we entered into a global licensing agreement with Apic Bio for AMT-162.

We have incurred $10.0 million of expenses related to the acquisition.

In addition, we have incurred $6.0 million of costs to initiate a Phase I/II clinical trial in 2024.

Huntington disease (AMT-130)

In the years ended December 31, 2024, 2023 and 2022, we incurred $18.0 million, $13.0 million and $19.8 million expenses respectively.

Our external costs for the development of AMT-130 were primarily related to the execution of our U.S.

and European studies.

The increase of $5.0 million in external cost in 2024 compared to 2023 is a result of enrolling patients into a third cohort between November 2023 and December 2024 as well as expenses we incurred associated with the RMAT application and in preparation of the Type B meeting with the FDA in November 2024.

The decrease of $6.8 million in external cost in 2023 compared to 2022 is a result of the completion of U.S.

patient enrollment in March 2022.

Temporal lobe epilepsy (AMT-260)

In the years ended December 31, 2023, December 31, 2022 and December 31, 2021, we incurred $13.0 million, $16.2 million and $0.9 million, respectively, expenses for the preclinical development of AMT-260, which we acquired on July 30, 2021.

In the years ended December 31, 2024, 2023 and 2022 we incurred $12.1 million, $13.0 million and $16.2 million expenses respectively.

In August 2023, the FDA cleared our IND application, and we started incurring costs for the preparation of a Phase I clinical trial.

The decrease in development costs in the year ended December 31, 2023 compared to 2022, results from completing the IND enabling studies, initiated during the year ended December 31, 2022, in the first half of 2023.

The increase in development cost in the year ended December 31, 2022, compared to 2021, related to costs incurred in relation to the toxicology study we initiated during the year as well as the manufacturing of supplies for clinical development.

Huntington disease (AMT-130)

We incurred $13.0 million, $19.8 million and $10.5 million expenses in the years ended December 31, 2023, 2022 and 2021 respectively.

Our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II(b) clinical trials in the U.S.

and in Europe.

We enrolled 26 patients into our U.S.

clinical trial between June 19, 2020 and March 21, 2022 and enrolled 13 patients into our European clinical trial between June 23, 2022 and June 21, 2023.

The decrease of $6.8 million in external cost in the year ended December 31, 2023 compared to 2022 is a result of enrolling less patients into the clinical trial during 2023, following completion of U.S.

enrollment in March 2022.

The increase of $9.3 million in external cost in the year ended December 31, 2022 compared to 2021 is a result of increased enrolling activities in 2022, including cross-over patients from our U.S.

trial and starting to enroll patients into our European trial in 2022, as well as follow-up activities related to patients enrolled in prior periods.

Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)

In January 2023, we entered into a global licensing agreement with Apic Bio for AMT-162.

In the years ended December 31, 2024 and 2023, we incurred $7.3 million and $6.0 million of expenses, respectively, to initiate a Phase I/II clinical trial in which enrolled the first patient in October 2024.

During the year ended December 31, 2023, we incurred expenses of $10.0 million related to the upfront consideration paid to Apic Bio for the global licensing rights to AMT-162.

Fabry disease (AMT-191)

In the years ended December 31, 2023, December 31, 2022 and December 31, 2021, we incurred $2.7 million, $2.9 million and $0.9 million expenses, respectively, primarily related to our preclinical activities.

In the years ended December 31, 2024, 2023 and 2022 we incurred $4.8 million, $2.7 million and $2.9 million expenses, respectively, related to our development of AMT-191.

In November 2023, the FDA cleared the IND application, and we started incurring additional costs for Phase I/II clinical trial preparation.

The decrease of $0.2 million in external costs in the year ended December 31, 2023 compared to 2022 is a result of completing the IND enabling studies by November 2023.

The increase of $2.0 million external costs in the year ended December 31, 2022 compared to 2021 is a result of initiating IND-enabling toxicology studies in August 2022.

Etranacogene dezaparvovec (AMT-060/061)

In the years ended December 31, 2023, December 31, 2022 and December 31, 2021, we incurred $1.4 million income, $2.5 million expenses and $8.7 million expenses, respectively which were primarily related to the Phase II development of the hemophilia B program.

After the Closing of the CSL Behring Agreement in May 2021, CSL Behring was responsible for the clinical and regulatory activities and commercialization of the Product.

We managed the existing trials on behalf of CSL Behring until such responsibilities were transitioned to CSL Behring in December 2022.

Direct research and development expenses related to clinical development incurred in the year ended December 31, 2022 and 2021 are presented net of reimbursements due from CSL Behring.

In the same periods , we also incurred costs related to the long-term follow-up of patients in our Phase I/II clinical trial of AMT-060 and our Phase IIb clinical trial of etranacogene dezaparvovec.

These costs are also presented net of reimbursements due from CSL Behring.

The decrease of $3.8 million in externals cost in the year ended December 31, 2023 compared to 2022 is a result of transitioning the activities to CSL Behring in December 2022 and winding down the support in early 2023.

The decrease of $6.3 million external cost in the year ended December 31, 2022 compared to 2021 is a result of CSL Behring being responsible from May 2021 onwards.

Costs incurred in the year ended December 31, 2022 primarily related to our preclinical activities.

Preclinical programs & platform development

In the years ended December 31, 2023, 2022 and 2021 we incurred $11.0 million, $7.2 million and $8.0 million, respectively, of costs related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

In the years ended December 31, 2024, 2023 and 2022 we incurred $3.9 million, $11.0 million and $7.2 million expenses, respectively, related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

Hemophilia B (AMT-061)

We transitioned activities related to the clinical trial and long-term follow-up of patients to CSL Behring in December 2022.

Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the years ended December 31, 2023 and 2022 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition.

Other research & development expenses

● We incurred $49.8 million in employee and contractor expenses in the year ended December 31, 2024 compared to $74.6 million in 2023 and $64.9 million in 2022.

The increase in 2023 of $11.8 million, compared to 2022, primarily related to the hiring of new personnel and contractors to support our clinical-stage programs and manufacturing operations as well as incurring $2.2 million in severance costs related to the Reorganization with no such cost incurred in 2022.

Our cost increased in 2022 by $9.2 million compared to 2021 primarily as a result of an increase in personnel and contractor related expenses to support the preclinical and clinical trial development of our product candidates;

● We incurred $29.4 million in operating expenses and depreciation expenses related to our rented facilities in the year ended December 31, 2023 compared to $23.6 million in 2022 and $18.8 million in 2021.

Our cost decreased in 2024 by $24.7 million compared to 2023 primarily as a result of the July 2024 Lexington Transaction as well as the October 2023 and August 2024 reorganizations.

The increase in 2023 of $9.6 million, compared to 2022, primarily related to the hiring of new personnel and contractors in 2022 through mid-2023 to support our clinical-stage programs and manufacturing operations;

● We incurred $21.6 million in operating expenses and depreciation expenses related to our rented facilities in Amsterdam and Lexington, Massachusetts in the year ended December 31, 2024 compared to $29.4 million in 2023 and $23.6 million in 2022.

The decrease in 2024 compared to 2023 of $7.9 million is primarily a result of the July 2024 Lexington Transaction.

Our costs increased by $5.9 million in 2023 compared to 2022 as a result of incurring additional operating and depreciation expenses in both our Lexington and Amsterdam facilities;

The increase in 2022 compared to 2021 of $4.8 million primarily related to operating expenses and depreciation expense incurred from additional sites in Lexington and increased depreciation expense related to the expansion of the Amsterdam facility in 2021;

● We incurred $13.2 million in disposables costs in the year ended December 31, 2023 compared to $17.8 million in the year ended December 31, 2022 and $14.7 million in the year ended December 31, 2021.

The decrease in 2023 related to the reduction in activities in the second half of 2023 due to our Reorganization.

The increase in 2022 related to the expansion of our activities to support the development of our product candidates;

● We incurred $9.3 million in share-based compensation expenses in the year ended December 31, 2024 compared to $16.9 million in 2023 and $18.4 million in 2022.

The decrease in 2023 of $1.5 million, compared to 2022, is primarily due to forfeitures as a result of the Reorganization and other severance, and a decrease in the fair value of awards granted.

This was partially offset by recognizing compensation cost related to performance share units granted in 2021, for which achievement of related performance conditions was deemed probable during the year ended December 31, 2023.

The increase in 2022 compared to 2021 of $5.6 million was primarily driven by the increase in awards granted, including those to newly recruited personnel as well as an increase in expense related to performance share units that were deemed probable;

● We incurred $15.9 million of expenses for the year ended December 31, 2023 related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to $7.1 million and $6.7 million for the same periods in 2022 and 2021.

The increase in 2023 was primarily due to an increase in the probability of making future milestone payments following the dosing of the first patient in Phase I/II clinical trial of AMT-260 after receiving IND acceptance in August 2023;

● We incurred $1.4 million of costs related to the impairment of the Lexington, MA facility right-of-use asset and related leasehold improvements for the year ended December 31, 2023 compared to nil in prior periods;

The decrease in 2024 of $7.6 million, compared to 2023, is primarily a result of the July 2024 Lexington Transaction, the reorganizations in October 2023 and August 2024 and a decrease in the fair value of the long-term incentive awards granted.

The decrease in 2023 of $1.5 million, compared to 2022, is primarily due to the October 2023 reorganization and a decrease in the fair value of long-term incentive awards granted.

This was partially offset by cost related to performance share units granted in 2021, for which achievement of related performance conditions was deemed probable in 2023 and 2024;

● We incurred $5.1 million in disposables costs in the year ended December 31, 2024 compared to $13.2 million in 2023 and $17.8 million in 2022.

The decrease in 2024 primarily related to the July 2024 Lexington Transaction as well as the reorganizations in October 2023 and August 2024.

The decrease in 2023 compared to 2022 was related to the October 2023 reorganization;

● We incurred $4.0 million of severance costs in the year ended December 31, 2024 related to the July 2024 Lexington Transaction and the August 2024 reorganization, compared to $2.1 million of severance cost in 2023 related to the October 2023 reorganization.

No such costs were incurred in 2022;

● We incurred nil and $1.4 million of costs in the years ended December 31, 2024 and 2023, respectively, related to the impairment of the Lexington, MA facility right-of-use asset and related leasehold improvements, compared to nil in 2022;

● We recognized a $1.8 million gain in the year ended December 31, 2024 related to a decrease in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to losses of $15.9 million in 2023 and $7.1 million in 2022.

The increase in the 2023 loss was primarily due to an increase in the probability of making future milestone payments due upon dosing of the first patient in Phase I/II clinical trial of AMT-260 after receiving IND acceptance in August 2023;

● We incurred $9.7 million in other expenses in the year ended December 31, 2024 compared to $6.8 million in 2023 and $17.2 million in 2022.

The decrease in costs in 2023 of $10.4 million, compared to 2022, is due to a decrease in contractual license expenses and a reduction in consultant-related expenses.

For the year ended December 31, 2023 we incurred $3.1 million in contractual license expenses upon the EMA approval of HEMGENIX® in February 2023 while for the year ended December 31, 2022, we incurred $7.0 million of contractual license expense upon FDA approval of HEMGENIX ® and $1.1 million contractual license expense for a valid patent claim granted within the EU.

The increase in 2022 compared to 2021 is primarily related to the contractual license expenses incurred in 2022.

The increase in costs in 2024 of $2.9 million, compared to 2023, is primarily a result of an increase in information technology expenses partially offset by decreases in contractual license expenses and consultant-related expenses.

The decrease in costs in 2023 of $10.4 million, compared to 2022, is due to decreases in contractual license expenses and consultant-related expenses.

Selling, general and administrative expenses

1 unchanged sentence

We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio.

Our selling costs include employee expenses as well as professional fees related to the preparation of a commercial launch of HEMGENIX® and advisory fees related to obtaining the CSL Behring Agreement.

Our selling costs included employee expenses as well as advisory fees related to obtaining the commercialization and license agreement (“CSL Behring Agreement”) between us and CSL Behring LLC (“CSL Behring”).

Selling, general and administrative expenses for the year ended December 31, 2024 were $52.7 million, compared to $74.6 million and $55.1 million for the years ended December 31, 2023 and 2022, respectively.

● We incurred $24.8 million in personnel and contractor expenses in 2023 compared to $21.1 million in 2022 and $16.0 million in 2021.

The increase in 2023 of $3.7 million, compared to 2022, and the increase in 2022 of $5.1 million, compared to 2021 was primarily related to the hiring of personnel and contractors to support our business operations.

In 2023, we also incurred $0.4 million in severance expense related to the Reorganization;

● We incurred $17.4 million of share-based compensation expenses in 2023 compared to $15.5 million in 2022 and $12.8 million in 2021.

The increase in 2023 compared to 2022 of $1.8 million was primarily related to an increase in awards granted and from recognizing compensation cost for performance share units granted in December 2021 that were deemed probable in the year ended December 31, 2023 offset by a decrease in expense related to a decrease in the fair value of awards granted.

The increase in 2022 compared to 2021 of $2.7 million was primarily related to the increase in awards granted, including those to newly recruited personnel as well as an increase in expense related to performance share units that were deemed probable;

● We incurred $11.7 million in professional fees in 2023 compared to $7.1 million in 2022 and $9.4 million in 2021.

Year ended December 31,

(in thousands)

Employee and contractor-related expenses

Share-based compensation expense

Professional fees

Depreciation and facility costs

Intellectual property fees

Severance costs

Information technology costs

Financial advisory fees

Other expenses

Total selling, general and administrative expenses

● We incurred $21.3 million in personnel and contractor expenses in the year ended December 31, 2024 compared to $24.4 million in 2023 and $21.4 million in 2022.

The decrease in 2024 of $3.1 million, compared to 2023, was primarily related to the reduction in personnel and contractors as a result of the Lexington Transaction and the October 2023 and August 2024 reorganizations.

The increase in 2023 of $3.3 million, compared to 2022, was primarily related to the hiring of personnel and contractors to support our business operations;

● We incurred $11.9 million of share-based compensation expenses in the year ended December 31, 2024 compared to $17.4 million in 2023 and $15.5 million in 2022.

The decrease in 2024 of $5.5 million, compared to 2023, was primarily related to the Lexington Transaction, the October 2023 and August 2024 reorganizations, and a decrease in the fair value of long-term incentive awards granted, partially offset by an increase in long-term incentive awards granted and costs associated with performance share units, granted in December 2021, for which achievement of performance criteria was deemed probable in 2024.

The increase in 2023 of $1.9 million, compared to 2022, was primarily related to an increase in long-term incentive awards granted and costs associated with performance share units, granted in December 2021, for which achievement of performance criteria was deemed probable in 2023.

These increases were partially offset by a decrease in expense related to a reduction in the fair value of long-term incentive awards granted;

● We incurred $7.3 million in professional fees in the year ended December 31, 2024 compared to $11.7 million in 2023 and $7.1 million in 2022.

We regularly incur accounting, audit and legal fees associated with operating as a public company.

In the year ended December 31, 2023, we incurred additional costs in professional fees related to our global licensing agreement with Apic Bio, entering into the Royalty Purchase Agreement, and other corporate initiatives.

Additionally, in the year ended December 31, 2021 we incurred professional fees in relation to our licensing transaction with CSL Behring and our acquisition of uniQure France SAS;

● We incurred $3.8 million, $1.0 million and $5.1 million in financial advisory fees in relation to our licensing transaction with CSL Behring in the years ended December 31, 2023, December 31, 2022 and December 31, 2021;

In 2024, we incurred additional professional fees related to the Lexington Transaction.

In 2023, we incurred additional professional fees related to our global licensing agreement with Apic Bio, the Royalty Purchase Agreement and other corporate initiatives;

● We incurred $1.7 million in intellectual property fees including registration and professional fees in the year ended December 31, 2024 compared to $3.8 million in 2023 and $1.5 million in 2022.

The increase in 2023 compared to 2022 of $2.3 million is mainly related to an increase in professional fees.

The decrease in 2022 compared to 2021 of $0.9 million related to a decrease in intellectual property license fees and registration costs;

● We incurred $11.6 million in other operating expenses in 2023 compared to $7.9 million in 2022 and $8.2 million in 2021.

The increase in 2023 compared 2022 of $3.7 million was primarily a result of an increase in information technology expenses.

The decrease of $2.1 million in 2024, compared to 2023, is primarily due to a decrease in professional fees.

The increase of $2.3 million in 2023, compared to 2022, is mainly related to an increase in professional fees;

● We incurred $1.2 million of severance costs in the year ended December 31, 2024 related to the Lexington Transaction and the August 2024 reorganization and $0.4 million of severance costs in 2023 related to the October 2023 reorganization.

No such costs were incurred in 2022;

● We incurred nil, $3.8 million and $1.0 million in financial advisory fees pertaining to our licensing transaction with CSL Behring in the years ended December 31, 2024, 2023 and 2022, respectively.

Other items, net

In the year ended December 31, 2023, we recognized $0.8 million in other expense in relation to a reduction in the fair market value of our equity stake in VectorY B.V.

In year ended December 31, 2024, we recognized a $1.2 million net gain related to the Lexington Transaction, with no such amounts recognized in 2023 and 2022.

We recognized $3.2 million in net other expenses during the year ended December 31, 2024 (nil in 2023 and 2022) related to the purchase of HEMGENIX® from Genezen, net of income from the sales of HEMGENIX® to CSL Behring, amortization of the intangible asset for our favorable supply terms under the CSA and credits from the release of liabilities related to expected net losses associated with minimum purchase commitments under the CSA.

We recognized nil income related to our equity stake in VectorY B.V.

in the year ended December 31, 2024.

In 2023, we recognized $0.8 million in other expense related to a reduction in the fair market value of our equity stake in VectorY B.V.

following an October 2023 financing round.

We recognized other income of $0.3 million and $3.0 million related to the equity stake for the years ended December 31, 2022 and 2021, respectively.

We received the equity stake in VectorY B.V.

in conjunction with a settlement agreement that the Company and VectorY B.V.

entered into in April 2021.

We recognized nil in other income of employee retention credit under the U.S.

CARES Act in the year ended December 31, 2023, compared to nil and $2.6 million of such income for the same periods in 2022 and 2021, respectively.

In 2023, we recognized $5.0 million in income related to payments received from European authorities to subsidize our research and development efforts in the Netherlands compared to $5.6 million in 2022 and $5.3 million in 2021.

Other income for the years ended December 31, 2023, 2022, and 2021 also includes income from the subleasing of a portion of our Amsterdam facility.

We recognized other income of $0.3 million related to the equity stake in 2022.

In 2024, we recognized $5.6 million in income related to payments received from European authorities to subsidize our research and development efforts in the Netherlands and France compared to $5.3 million in 2023 and $6.1 million in 2022.

Other income for the years ended December 31, 2024, 2023, and 2022 also includes income from the subleasing of a portion of our Amsterdam facility and Lexington research and development facility.

We present expenses related to such income as other expense.

4 unchanged sentences

Interest income

Interest expense

Interest expense - Royalty Financing Agreement

Interest expense - Hercules debt facility

Foreign currency (losses) / gains, net

Other non-operating gains / (losses)

Other non-operating gains

Total non-operating (expense) / income, net

We recognize interest income associated with our cash and cash equivalents and investment securities.

We recognized $19.6 million interest income in 2023, $0.6 million in 2022 and $0.2 million in 2021.

Interest income increased by $19.0 million in 2023 compared to 2022 as a result of earning interest income from investing the proceeds of our May 2023 Royalty Financing Agreement as well as the $100.0 million milestone payment collected from CSL Behring in July 2023 into debt securities.

In addition, our interest income in 2023 and 2022 compared to 2021 benefited from an increase in interest rates on cash on hand.

Our interest income increased in 2022 by $0.4 million compared to 2021 primarily due to the interest income earned on investment securities.

We recognized $41.6 million interest expense in 2023, $11.7 million in 2022 and $7.5 million in 2021.

Interest expense increased by $29.9 million in 2023 compared to 2022 due to an increase in market interest rates related to the Hercules debt as well as the recognition of $26.9 million of accrued interest expense related to the Royalty Financing Agreement that we entered into in May 2023.

Our interest expense in 2022 primarily increased by $4.2 million compared to 2021 due to an increase in market interest rates in 2022.

We recognized interest income of $21.4 million in 2024, $19.6 million in 2023 and $0.6 million in 2022.

Our interest income increased in 2024 by $1.9 million compared to 2023 primarily due to the interest income earned on investment securities and cash on hand.

Interest income increased by $19.0 million in 2023, compared to 2022, as a result of receiving $370.1 million of net proceeds from the May 2023 royalty financing agreement and a $100.0 million milestone payment from CSL Behring in July 2023.

We recognized non-cash interest expenses related to the May 2023 royalty financing agreement of $50.9 million in 2024, $26.9 million in 2023 and nil in 2022.

We recognized interest expense of $12.9 million in 2024, $14.6 million in 2023 and $11.7 million in 2022 related to the Hercules debt facility.

Interest expense decreased by $1.7 million in 2024, compared to 2023, primarily due to the $50.0 million repayment of Hercules debt in July 2024, as well as a decrease in market interest rates.

Interest expense increased by $2.9 million in 2023 compared to 2022 due to an increase in market interest rates related to the Hercules debt.

We hold monetary items and enter into transactions in foreign currencies, predominantly in euros and U.S.

We recognize foreign exchange results related to changes in these foreign currencies.

In 2023, we recognized a net foreign currency loss of $1.7 million related to our borrowings from Hercules, the Royalty Financing Agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group, compared to a net gain of $23.2 million in 2022 and a net gain of $29.7 million in 2021.

In 2023, we recognized nil within Other non-operating gains /(losses).

In 2022, we recognized a $2.8 million net gain within Other non-operating gains / (losses) related to a decrease in the fair value market value of a derivative financial liability related to the change of control payment (“CoC-payment”) compared to a net loss of $0.2 million in 2021.

We recorded a net gain in 2022 as no change of control event had occurred as of the February 22, 2023 Termination Date of the amended BMS CLA.

We had recorded a net loss of $0.2 million in 2021 for the increase in the fair market value of the derivative financial liability related to the CoC-payment.

We recognized $1.9 million of deferred tax expense in 2023, compared to $1.5 million of deferred tax income in 2022 and $3.2 million of deferred tax expense in 2021.

In 2023, deferred tax expense recorded in the U.S., related to the consumption of net operating losses, more than offset the deferred tax income recorded as a result of the buildup of net operating losses by the French entity.

Deferred tax income recorded in 2022 results from deferred tax benefits recorded related to the buildup of net operating losses by the French entity which are partially offset by deferred tax expense recorded in the U.S.

as a result of the consumption of net operating losses.

In addition, we recorded deferred tax expense resulting from the release of valuation allowance for the tax benefit of share issuance costs within the Netherlands in 2022.

Deferred tax expense recorded in 2021 results from the consumption of net operating tax losses by our U.S.

entity as well as deferred tax expense resulting from the release of valuation allowance for the tax benefit of share issuance costs within the Netherlands.

In 2024, we recognized a net foreign currency loss of $10.5 million related to our borrowings from Hercules, the May 2023 royalty financing agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group.

We recognized a net loss of $1.7 million in 2023 and a net gain of $23.2 million in 2022.

In 2024 and 2023, we recognized no Other non-operating gains.

In 2022, we recognized a $2.8 million net gain related to a decrease in the fair value market value of a derivative financial liability

We recognized $2.3 million of deferred tax expense in 2024 compared to $1.9 million of deferred tax expense in 2023 and $1.5 million of deferred tax income in 2022.

In 2024 and 2023 deferred tax expense recorded in the U.S., related to the consumption of net operating losses.

From 2022 up to mid-2023 we recognized deferred tax income in France as a result of the buildup of net operating losses by the French entity.

The deferred tax income in France in 2024 is almost fully offset with an increase in valuation allowance.

Financial Position, Liquidity and Capital Resources

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Until such time, if ever, as we can generate substantial cash flows from successfully commercializing our proprietary product candidates, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances and marketing, distribution, and licensing arrangements.

Based on our current operating plan, research and development plans and our timing expectations related to the progress of our programs and following the Reorganization, we believe that our cash and cash equivalents and investment securities will fund our operations into second quarter of 2027.

Based on our current operating plan, research and development plans and our timing expectations related to the progress of our programs, we believe that our cash and cash equivalents and investment securities will fund our operations through the second half of 2027.

We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect.

We expect that we will require additional funding if we decide to advance AMT-130 for our Huntington’s disease gene therapy program or any of our other product candidates into late-stage clinical development.

The amount and timing of our actual expenditures may vary significantly depending on numerous factors, including the timing of a potential BLA submission, approval and commercialization of AMT-130.

We expect that we will require additional funding if we decide to advance any of our other clinical product candidates into late-stage clinical development.

Our material cash requirements include the following contractual and other obligations:

As of December 31, 2023, we had an outstanding loan amount owed to Hercules for an aggregate principal amount of $100.0 million.

As of December 31, 2024, we had an outstanding loan amount owed to Hercules Capital, Inc.

(“Hercules”) for an aggregate principal amount of $50.0 million.

In July 2024, in connection with the Closing of the Lexington Transaction, we repaid $50.0 million of the $100.0 million of principal outstanding as well as $3.1 million in end-of-term fees.

Future interest payments and financing fees associated with the loan total $16.0 million, with $8.6 million payable within 12 months.

We are contractually required to repay the $100.0 million in full in January 2027.

We entered into lease arrangements for facilities, including corporate, manufacturing and office space.

We are contractually required to repay the $50.0 million principal outstanding in full in January 2027.

We entered into lease arrangements for facilities, including corporate, laboratory and office space.

As of December 31, 2024, we had fixed lease payment obligations of $23.0 million, with $4.1 million payable within 12 months.

Following the Closing of the Lexington Transaction, we assigned our lease for the Lexington facility to Genezen.

This reduced our fixed lease payment obligations by $20.7 million.

We continue to guarantee such payments until the end of the lease term in May 2029.

Commitments related to acquisition of uniQure France SAS (nominal amounts)

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In September 2023, we made a payment of EUR 10.0 million ($10.6 million) to the former shareholders of uniQure France SAS following the FDA’s clearance of the IND application for AMT-260.

As of December 31, 2023, our remaining commitment amounts include a EUR 30.0 million ($33.1 million) milestone payment due upon treating the first patient in a Phase I/II clinical trial for AMT-260 and EUR 160.0 million ($176.6 million) in potential milestone payments associated with Phase III development and the approvals of AMT-260 in the U.S.

In December 2024, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France SAS following the dosing of the first patient in Phase I/II clinical trial for AMT-260.

As of December 31, 2024, our remaining commitment amounts include a EUR 160.0 million ($166.2 million) in potential milestone payments associated with Phase III development and the approvals of AMT-260 in the U.S.

and European Union.

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These payments are owed in euro and have been translated at the foreign exchange rate as of December 31, 2024, of $1.04/€1.00.

As of December 31, 2023, we expect these obligations will become payable between the first half of 2024 and 2031.

As of December 31, 2024, we expect these obligations will become payable between 2029 and 2033.

If and when due, up to 25% of the milestone payments can be settled with our ordinary shares.

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We also owe payments to a financial advisor related to certain payments we will collect under the CSL Behring Agreement.

Commitments related to the CSA and DMSA with Genezen

Our CSA with Genezen requires us to take or pay contract manufacturing services for HEMGENIX® during a three-year period ending July 2027.

As of December 31, 2024, our remaining minimum purchase commitments to Genezen amount to $36.5 million, with $15.8 million to be paid within the next 12 months.

CSL Behring’s minimum purchase commitments to us over the next 12 months are $13.6 million.

Our DMSA with Genezen requires us to take or pay contract development services during a three-year period ending July 2027.

As of December 31, 2024 our remaining minimum purchase commitments amount to $12.5 million with $4.0 million to be paid within the next 12 months.

The table below summarizes our consolidated cash flow data for the years ended December 31:

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Net cash used in operating activities

Net cash used in investing activities

Net cash generated from financing activities

Net cash generated from / (used in) investing activities

Net cash (used in) / generated from financing activities

Foreign exchange impact

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in 1998, with the exception of generating income in 2021 after receiving the upfront payment upon Closing of the CSL Behring Agreement.

We continue to incur losses in the current period.

We recorded a net loss of $308.5 million for the year ended December 31, 2023, and net loss of $126.8 million in 2022, and a net income of $329.6 million in 2021.

We continued to incur losses in the current period.

We recorded a net loss of $239.6 million for the year ended December 31, 2024, and net loss of $308.5 million in 2023, and a net loss of $126.8 million in 2022.

As of December 31, 2024, we had an accumulated deficit of $1,130.0 million.

Sources of liquidity

From our first institutional venture capital financing in 2006 through the current period, we funded our operations primarily through private and public placements of equity securities, debt securities, payments from our collaboration partners as well as from selling a portion of royalties due from our collaboration partner CSL Behring.

In May 2021, we received a $462.4 million cash payment due from CSL Behring.

We have collected $55.0 million related to CSL Behring’s global regulatory submissions for etranacogene dezaparvovec in March and April 2022, and $100.0 million in July 2023 related to the first sale milestone of HEMGENIX® in the U.S., and are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from the Royalty Financing Transaction) on net sales of HEMGENIX®.

On March 1, 2021, we entered into a Sales Agreement with SVB Leerink LLC (“SVB Leerink”) with respect to an at-the-market (“ATM”) offering program, under which we, from time to time in our sole discretion, could offer and sell through SVB Leerink, acting as agent, our ordinary shares, up to an aggregate offering price of $200.0 million.

In the year ended December 31, 2021, we received net proceeds of $29.6 million from the issuance of 921,730 ordinary shares under the Sales Agreement that took place during March and April of that year.

We paid SVB Leerink a commission equal to 3% of the gross proceeds of the sales price of all ordinary shares sold through it as a sales agent under the Sales Agreement.

We did not issue any ordinary shares under the Sales Agreement for the 12-month periods ended December 31, 2023 and December 31, 2022.

On January 29, 2021, we drew down $35 million under a facility agreement with Hercules.

We drew down a further $30 million under our 2021 Restated Facility with Hercules in December 2021.

We and Hercules amended the 2021 Restated Facility on May 12, 2023.

The 2023 Amended Facility extends the maturity date and interest-only period from December 1, 2025 to January 5, 2027.

We are required to repay the entire principal balance of $100.0 million on the maturity date.

From our first institutional venture capital financing in 2006 through the current period, we funded our operations primarily through private and public placements of equity securities, debt securities, payments from our collaboration partners as well as $370.1 million from selling a portion of royalties due from our collaboration partner CSL Behring in 2023.

Between July 2021 and July 2023, we have collected $617.4 million from CSL Behring as a result of the sale of HEMGENIX® to CSL and other milestones collected from CSL, and we are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from the Royalty Financing Transaction) on net sales of HEMGENIX®.

On May 12, 2023 we and Hercules amended our debt facility.

The amendment extends the Maturity Date and interest-only period from December 2025 to January 2027.

In connection with the Closing of the Lexington Transaction in July 2024 we amended the debt facility again and repaid $50.0 million of the principal balance outstanding.

We are required to repay the remaining principal balance of $50.0 million on the Maturity Date.

The interest rate is adjustable and is the greater of (i) 7.95% and (ii) 7.95% plus the prime rate less 3.25% per annum.

Under the 2023 Amended Facility, we owe a back-end fee of $4.9 million on December 1, 2025 and a back-end fee of $1.3 million on January 5, 2027.

We are subject to certain covenants under the 2023 Amended Facility and may become subject to covenants under any future indebtedness that could limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends, which could adversely impact our ability to conduct our business.

We owe a back-end fee of $2.4 million on December 1, 2025 and a back-end fee of $0.6 million on the Maturity Date.

We are subject to certain covenants under the debt facility and may become subject to covenants under any future indebtedness that could limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends, which could adversely impact our ability to conduct our business.

In addition, our pledge of assets as collateral to secure our obligations under the 2024 Amended Facility may limit our ability to obtain debt financing.

The 2023 Amended Facility permits us to issue up to $500.0 million of convertible debt.

The debt facility permits us to issue up to $500.0 million of convertible debt.

To the extent we need to finance our cash needs through equity offerings or debt financings, such financing may be subject to unfavorable terms including without limitation, the negotiation and execution of definitive documentation, as well as credit and debt market conditions, and we may not be able to obtain such financing on terms acceptable to us or at all.

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If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves, which could have a material adverse effect on our business, financial conditions, results of operations and cash flows.

Net Cash used in / generated from operating activities

Net Cash used in operating activities

Year ended December 31,

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Cash flows from operating activities

Net (loss) / income

Adjustments to reconcile net (loss) /income to net cash (used in) / generated from operating activities:

Adjustments to reconcile net loss to net cash used in operating activities:

Depreciation, amortization and impairment

Amortization of premium/discount on investment securities

Amortization of discount on investment securities

Share-based compensation expense

Royalty financing agreement interest expense

Royalty financing agreement interest expense, net of interest paid

Deferred tax expense / (income)

Change in fair value of contingent consideration and derivative financial instrument, net

Unrealized foreign exchange (gains) / losses, net

Unrealized foreign exchange losses / (gains), net

Other items, net

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Contingent consideration milestone payment

Net cash (used in) / generated from operating activities

Net cash used in operating activities was $145.9 million for the year ended December 31, 2023, and consisted of a net loss of $308.5 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $11.9 million, amortization of the premium/discount on investment securities of $10.9 million, share-based compensation expense of $35.1 million, $26.9 million of interest expense related to the royalty financing agreement, a change in deferred taxes of $1.9 million, $15.9 million change in the fair value of contingent consideration and unrealized foreign exchange gains of $2.2 million.

Net cash used in operating activities

Net cash used in operating activities was $182.7 million for the year ended December 31, 2024, and consisted of a net loss of $239.6 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $12.6 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $22.3 million, $44.2 million of interest expense net of interest paid related to the May 2023 royalty financing agreement, a change in deferred taxes of $2.4 million, $1.8 million change in the fair value of contingent consideration and unrealized foreign exchange losses of $15.4 million.

Net cash generated from operating activities also included unfavorable changes in operating assets and liabilities of $3.9 million.

There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $2.2 million.

There was an decrease in inventory balances of $2.4 million.

There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $4.1 million, primarily related to a increase of $1.5 million in accounts payable and a decrease of $5.6 million related to various accruals.

Net cash used in operating activities also includes a payment for a contingent consideration milestone of $19.6 million.

Net cash used in operating activities was $145.9 million for the year ended December 31, 2023, and consisted of a net loss of $308.5 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $11.9 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $35.1 million, $25.6 million of interest expense net of interest paid related to the May 2023 royalty financing agreement, a change in deferred taxes of $1.9 million, $15.9 million change in the fair value of contingent consideration and unrealized foreign exchange gains of $2.2 million.

Net cash generated from operating activities also included favorable changes in operating assets and liabilities of $80.5 million.

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These changes also relate to a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $12.6 million, primarily related to an increase in accounts payable.

Net cash generated from operating activities was $288.0 million for the year ended December 31, 2021, and consisted of net income of $329.6 million adjusted for non-cash items, including depreciation and amortization expense of $7.3 million, share-based compensation expense of $25.6 million, a change in fair value of contingent consideration of $6.8 million, unrealized foreign exchange gains of $31.3 million, a change in deferred taxes of $3.2 million and other non-cash items, net, of $2.8 million.

Net cash generated from operating activities also included unfavorable changes in operating assets and liabilities of $50.3 million, which includes $55.0 million recognized as a contract asset related to probable CSL Behring milestone payments.

Additionally, these changes also related to a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $4.0 million primarily related to an increase in various prepaids, including those related to clinical trials, partially offset by decrease in receivables as a result of collection of the BMS milestone that was recorded as of December 31, 2020 and collection of the CSL Behring receivables recorded as of December 31, 2020 for expenses for which we had a right of reimbursement and a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $8.5 million primarily related to an increase in various accruals for goods received from and services provided by vendors and an increase in personnel accrual s.

Net income primarily consisted of $462.4 million license revenue recognized on Closing and $55.0 million license revenue related to CSL Behring’s global regulatory submissions for etranacogene dezaparvovec that occurred in March and April 2022 and were considered probable on December 31, 2021.

Net cash used in investing activities

In 2023, we used $205.7 million in our investing activities compared to $182.7 million in 2022 and $67.4 million in 2021.

Net cash generated from / (used in) investing activities

In 2024, we generated $163.0 million from our investing activities compared to using $205.7 million in 2023 and $182.7 million in 2022.

Year ended December 31,

(in thousands)

Investment in investment securities

Proceeds from maturity of investment securities

Build out of Amsterdam site

Build out of Lexington site

Proceeds from maturity of debt securities

Investment in debt securities

Divestment of commercial manufacturing facility

Capital expenditures - European sites

Capital expenditures - Lexington site

Acquisition of uniQure France SAS, net of cash acquired

Net cash used in investing activities

In the years ended December 31, 2023 and 2022, we invested $366.4 million and $163.1 million, respectively, of our cash on hand into euro and dollar denominated government bonds.

We made no such investments in 2021.

In the year ended December 31, 2023 we received $167.9 million in proceeds from the maturing of investments securities.

In 2023, we invested $3.4 million in the build out of our Amsterdam site compared to $11.9 million in 2022 and $12.4 million in 2021.

Our investments in 2023 and 2022 related to investments into equipment while in 2021 investments primarily related to the construction of additional laboratories to support the expansion of our research and development activities as well as the construction of a cleanroom designed to be capable of manufacturing cGMP materials at a 500-liter scale.

In 2023, we invested $3.8 million in our facility in Lexington compared to $5.8 million in 2022 and $5.0 million in 2021.

Our investments in 2023 are a result of improvements made to the lease facility and investment in office and laboratory equipment.

We paid EUR 1.8 million ($1.9 million) to acquire the remaining outstanding shares of uniQure France SAS in February, July and September 2022.

We paid EUR 42.1 million ($49.9 million), net of EUR 2.8 million ($3.3 million) of cash acquired, during the year ended December 31, 2021 to acquire 97.7% of the outstanding ordinary shares of uniQure France SAS on July 30, 2021.

Net cash generated from financing activities

Net cash generated from / (used in) investing activities

Net cash (used in) / generated from financing activities

Year ended December 31,

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Proceeds from Royalty Financing Agreement, net of debt issuance costs

Contingent consideration milestone payment

Proceeds from issuance of shares related to employee stock option and purchase plans

Proceeds from loan increment, net of debt issuance costs

Proceeds from issuance of ordinary shares, net of issuance costs

Repayment of debt assumed through the acquisition of uniQure France SAS

Net cash generated from financing activities

In June 2023, we received $370.1 million net proceeds from the Royalty Financing Agreement.

In September 2023, following the FDA’s clearance of the IND application for AMT-260, we made a payment of $10.6 million to the former shareholders of uniQure France SAS based on contractually defined milestones.

$9.6 million of this payment related to a contingent consideration of which $7.6 million was classified as cash flows from financing activities and $1.9 million was classified as a net cash flow used in operating activities.

In 2023, we received $0.3 million from the exercise of options to purchase ordinary shares issued in accordance with our share incentive plans, compared to $1.4 million in 2022 and $2.8 million in 2021.

In January 2021, we received $34.6 million net proceeds from the 2021 Amended Facility and in December 2021 we received $29.5 million net proceeds from the 2021 Restated Facility for combined net proceeds of $64.1 million (nil in 2023 and 2022).

We received net proceeds of $29.6 million associated with our ATM offering in March and April 2021.

No such proceeds were received in 2023 or 2022.

Upon the acquisition of uniQure France SAS, uniQure France SAS held loans with an outstanding amount equal to EUR 1.4 million ($1.6 million).

During the year ended December 31, 2021, the loans were repaid in their entirety.

Repayment of long-term debt

Contingent consideration milestone payment

Net cash (used in) / generated from financing activities

In December 2024, following the dosing of the first patient in Phase I/II clinical trial for AMT-260, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France SAS based on contractually defined milestones.

EUR 26.8 million ($28.2 million) of this payment related to a contingent consideration of which EUR 8.2 million ($8.6 million) was classified as cash flows from financing activities and EUR 18.7 million ($19.6 million) was classified as a net cash flow used in operating activities.

In September 2023, following the FDA’s clearance of the IND application for AMT-260, we made a payment of EUR 10.0 million ($10.7 million) to the former shareholders of uniQure France SAS based on contractually defined milestones.

EUR 8.9 million ($9.6 million) of this payment related to a contingent consideration of which EUR 7.2 million ($7.6 million) was classified as cash flows from financing activities and EUR 1.8 million ($1.9 million) was classified as a net cash flow used in operating activities.

Funding requirements

Our future capital requirements will depend on many factors, including but not limited to:

● contractual milestone payments and royalties we might be owed in accordance with the CSL Behring Agreement;

● activities related to submission of a BLA for AMT-130 in Huntington’s disease;

● activities to prepare for the commercialization of AMT-130 in Huntington’s disease in the United States;

● investments to launch AMT-130 in Huntington’s disease in the United States including the amounts of revenue we generate following the launch;

● earnout payments we might owe the former shareholders of uniQure France SAS, which are subject to the achievement of specific development and regulatory milestones;

● the scope, timing, results, and costs of our current and planned clinical trials, including those for AMT-130 in Huntington’s disease;

● contractual milestone payments and royalties we might be owed in accordance with the CSL Behring Agreement;

● the scope, timing, results, and costs of our current and planned clinical trials;

● the scope, obligations and restrictions on our business related to our existing equity, debt or royalty monetization financings and underlying agreements;

● the extent to which we acquire or in-license other businesses, products, product candidates or technologies;

● the amount and timing of revenue, if any, we receive from manufacturing products for CSL Behring;

● the scope, timing, results and costs of preclinical development and laboratory testing of our additional product candidates;

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● the costs and timing of preparing, filing, expanding, acquiring, licensing, maintaining, enforcing, and prosecuting patents and patent applications, as well as defending any intellectual property-related claims.

● the costs associated with maintaining quality compliance and optimizing our manufacturing processes, including the operating costs associated with our Lexington, Massachusetts manufacturing facility.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 7. Management’s Discussion and Analysis